CRISPR/Cas9-mediated correction of mutated copper transporter ATP7B

Wilson's disease (WD) is a monogenetic liver disease that is based on a mutation of the ATP7B gene and leads to a functional deterioration in copper (Cu) excretion in the liver. The excess Cu accumulates in various organs such as the liver and brain. WD patients show clinical heterogeneity, whi...

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Pubblicato in:PLoS One
Autori principali: Pöhler, Michael, Guttmann, Sarah, Nadzemova, Oksana, Lenders, Malte, Brand, Eva, Zibert, Andree, Schmidt, Hartmut H., Sandfort, Vanessa
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science 2020
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Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7526882/
https://ncbi.nlm.nih.gov/pubmed/32997714
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0239411
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